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Ampligen On the Clock: Hemispherx's 'Complete Response' Means Ball is Now in FDA's Court Now

[caption id="attachment_12929" align="alignright" width="325" caption="By end the January, 2013, at the latest,we will know if the FDA will approve Ampligen for ME/CFS..."]
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Moving quickly, Ampligen's producer, Hemispherx Biopharma, filed its 'complete response' to the FDA's request for data just 53 days after the agency agreed to relax its requirements for review of the drug. Hemispherx's 'complete response' means we will know by the end of January and perhaps sooner whether Ampligen becomes the first FDA approved drug for chronic fatigue syndrome (ME/CFS).

New Data

In its response Hemispherx provided new data derived from a 24 week extension period occurring after the 40 week original trial. Hemispherx stated the data will show that patients who remained on the drug for a further 24 weeks showed further improvements in endurance (23% - 39% increase in treadmill duration) suggesting that the drug's effectiveness increases over time.

Similarly, Hemipherx reported that patients who responded well to Ampligen showed significantly greater improvement relative to non-responders in areas such as vitality, general health, Karnofsky performance (functionality) and activities of daily living the longer they were on the drug. This is important because the original study did not highlight improvements in functionality and well-being. This data suggests Ampligen works 'globally' to improve both endurance and functionality.

FDA and Ampligen

It appears that the FDA has at least several possibilities;
  • it can approve the drug
  • it can reject the drug
  • it can give the drug 'accelerated approval' status - in this scenario Ampligen is not approved but Hemispherx is allowed to market the drug and gather more data for a final attempt at approval
  • it can ask Hemispherx for more data - without the income derived from 'accelerated approval' this outcome would be difficult for Hemispherx.
Hemispherx's Press Release suggests the company is gunning for accelerated approval status. While the FDA did recently substantially relax its requirements to consider Ampligen for approval its possible, perhaps even probable, they still want that large, multi-dose, placebo-controlled, double-blinded treatment trial originally required in their 2009 report. Hemispherx didn't come anywhere near providing that kind of data but getting accelerated approval would give them the means to do so.

The FDA has increasingly come under fire to approve more drugs and, in particular, to approve more drugs for chronic illnesses and the new law provided drug companies an opening to do just that.

New Law Presents Opportunity for Hemispherx and Ampligen

In their press release Hemispherx highlighted the fact that the 2012 Food and Drug Safety and Innovation Act (FDSIA) law encourages the FDA to take 'innovative and flexible approaches' to assessing treatment for 'serious' disorders with 'unmet' needs.

In section 901 from the FDASIA Act below note the emphases on 'expedited...review' and the need to target subpopulations.

"the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions... This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation
Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.​

Indeed, recent history suggests a drug need be effective in only a small group of patients if if few treatment options are present. With FDA analyses suggesting that 11 lupus patients needed to be treated on Benylstra to find one the drug helped, and with the drug showing marginal effectiveness overall, but with the last FDA approved drug for lupus dating back 50 years, Benylstra was easily approved. (Benlystra significant helped those few patients which responded.)

Hemispherx's production facility at New Brunswick will be undergoing 'pre-approval inspection' as part of the FDA review process. If the drug is approved or granted accelerated approval status Hemispherx should, hopefully, be able to hit the ground running. Could the drug be widely available to ME/CFS patients sometime next year? Time will tell.

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Comments

I don't think Ampligen is the answer. It's not if you believe that ME/CFS has an autoimmune component or that it causes neuroinflammation (and ME definitely does cause neuroinflammation). If you look up the drug under its brand name, none of this info will come to light, but if you go to pubmed and look it up under its chemical name (Poly I:C, or poly-inosinic polycytidilic acid), you'll find lots of hugely discouraging studies, some of which even call Ampligen "neurotoxic."
I'm not saying this to be discouraging. I want Ampligen to work as much as anyone -- I suffer. But I think there's a reason Ampligen wasn't rushed into duty two decades ago and I think the ME/CFS community might be better served going in other pharmaceutical directions.

http://www.ncbi.nlm.nih.gov/pubmed/18590811

http://www.ncbi.nlm.nih.gov/pubmed/21123556

http://www.ncbi.nlm.nih.gov/pubmed/21296697
http://www.ncbi.nlm.nih.gov/pubmed/21815968
 
Reply to grosolo,

Sorry grosolo, but your information is missing a vital component. Ampligen is not simply Poly I:C as described in the links you presented. Ampligen is a specifically modified form of Poly I:C that retains TLR3 activity but is designed to have a much shorter half-life in the body.
http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=/netahtml/PTO/srchnum.htm&r=1&f=G&l=50&s1=4,024,222.PN.&OS=PN/4,024,222&RS=PN/4,024,222

This results in much less toxicity than Poly I:C. The toxicity of Poly I:C was known years before Ampligen because it was researched as an immune stimulant to fight cancer. It proved too toxic for even that use, and Ampligen was invented as a less toxic immune stimulant. Ampligen proved to be very efficacious in stimulating the TLR3 interferon system to fight viruses and also has some anticancer activity.
 
Skepticism about whether doctors or insurance companies will prescribe the drug is a bit premature at this point, the first step is getting the drug approved in the first place.

Getting a drug approved for CFS or ME sets a major precedent and will benefit us even if we don't ever take it personally.
 
I don't think Ampligen is the answer. It's not if you believe that ME/CFS has an autoimmune component or that it causes neuroinflammation
The apparent autoimmunity and neuroinflammation in ME/CFS are likely caused by one or more infections. Enteroviruses, for example, which are strongly linked to ME/CFS, are inducers of autoimmunity. 1 In general, infections precipitate the production of lots of autoantibodies.

So by wiping out viral infections with Ampligen, or with some other antiviral developed in the future, you will likely halt the autoimmunity.
 
I don't think Ampligen is 'the answer'; I think it's like part of an answer; pushing forward to a bigger answer...Its definitely a big help....a big big help for some people...a smaller help for others and it doesn't work for others but that's what you'd expect from anything that works in this disorder - until subsets are teased out.
 
The apparent autoimmunity and neuroinflammation in ME/CFS are likely caused by one or more infections. Enteroviruses, for example, which are strongly linked to ME/CFS, are inducers of autoimmunity. 1 In general, infections precipitate the production of lots of autoantibodies.

So by wiping out viral infections with Ampligen, or with some other antiviral developed in the future, you will likely halt the autoimmunity.
If the medicine in question causes brain toxicity -- even while stimulating the immune system -- it's still a very flawed treatment. Look up Poly inosinic poly cytidilic acid on pubmed. Scientists now administer Ampligen to mice in order to test other medicines under the conditions of neurotoxicity. IE -- they're using Ampligen to ruin the brains of mice in order to see how other drugs can fix the brain. That says a lot...And these are the non-Hemispherx researchers -- the ones who have no stake in this...

I don't mean to be a kill-joy. I want everyone to be able to benefit from whatever they can. But there has been so much research that has been glossed over. The whole brand name Ampligen, not to mention the recently made-up fake generic name rintatolimod -- which no one uses -- are modes of obfuscation. Because if you look up the drug under that name, you get very little information. But if you look up studies under its chemical name of poly inosinic poly cytidilic acid, you get so much more...and a lot of it is ugly and scary.
 
Reply to grosolo,

Sorry grosolo, but your information is missing a vital component. Ampligen is not simply Poly I:C as described in the links you presented. Ampligen is a specifically modified form of Poly I:C that retains TLR3 activity but is designed to have a much shorter half-life in the body.
http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=/netahtml/PTO/srchnum.htm&r=1&f=G&l=50&s1=4,024,222.PN.&OS=PN/4,024,222&RS=PN/4,024,222

This results in much less toxicity than Poly I:C. The toxicity of Poly I:C was known years before Ampligen because it was researched as an immune stimulant to fight cancer. It proved too toxic for even that use, and Ampligen was invented as a less toxic immune stimulant. Ampligen proved to be very efficacious in stimulating the TLR3 interferon system to fight viruses and also has some anticancer activity.
This patent form just says that polyriboinosinate and polycytidilate were modified to YIELD Poly IC.
So they didn't modify Poly IC to create a less toxic form. They modified other drugs to create Poly IC. So anytime you see a study coming out now that uses Poly IC, it's using Ampligen, plain and simple...